Patient-Centered Outcomes in Infants and Young Children with Cleft Lip and Palate

 描述(申请人提供)：制定终点策略，包括父母报告的评估婴幼儿临床试验干预措施的措施 这些活动包括：1)评估唇裂伴或不伴腭裂(CL±P)；2)对照现有临床结果评估，评估已开发的亲代措施的测量模型、可靠性和广告有效性；3)评估亲代措施发现与初次唇裂修复相关的改善的能力；4)在现场领导者的指导下，最终确定终点策略，并在临床试验中实施亲代措施。意义：患有CL±P的婴儿和儿童经常在进食、营养、听力、言语产生、呼吸和睡眠方面遇到重大挑战。出于这个原因，从出生到18个月大，他们经常接受许多手术和非手术干预，包括正畸塑造嘴唇和鼻子，修复唇裂和腭裂，以及放置鼓室造口管。然而，尽管公布了护理指南，但国内和国际上对患有CL±P的婴儿的护理存在巨大差异。这在一定程度上是因为缺乏标准的结果衡量标准，可以适用于这个年龄段的各种治疗。创新：该项目将有助于先天性CL±P患者一生中结果的发展，并将提供一个终点战略，以补充两个最大的唇裂财团--American Left和Eurocleft的努力。我们建议开发新的结果来衡量父母报告的可观察结果。FDA正在探索这些方法，用于设备和药物开发中的合格仪器。该项目将评估与该领域领导者商定的临床评估相关的母体措施的测量属性。我们在临床试验中处理整个终点策略，并以患者为中心扩大现有措施，这是NIH研究的高度优先事项。该项目的潜在影响很高，因为我们参与了美国左翼和欧洲分裂组织领导人的参与，以及我们计划让来自美国和海外的该领域领导人参与我们开发的端点战略的评估。方法与分析：我们将与家长和专家小组进行第一阶段访谈和焦点小组，以获得关于家长测量内容和临床评估选择的共识。在第二阶段，我们将评估父母措施的测量性质，并与专家小组召开会议，最终确定父母措施和终点战略，包括在临床试验中实施父母措施。研究人员：一支由经验丰富的研究人员和临床医生(儿科医生、外科医生、临床心理学家、临床社会工作者、生物统计学家、成果科学家)组成的具有合作历史的多学科团队能够很好地成功实现研究目标。

 DESCRIPTION (provided by applicant): Develop an endpoint strategy, including parent-reported measures for evaluation of interventions in clinical trials for infants and young children with cleft lip with or without cleft palate (CL± P), 2) Evaluate measurement model, reliability, ad validity of developed parent measures in relation to existing clinical outcome assessments, 3) Evaluate the ability of parent measures to detect improvements associated with primary cleft repair, 4) Finalize the endpoint strategy and implement parent measures in clinical trials with guidance from field leaders. Significance: Infants and children with CL± P often experience significant challenges with feeding, nutrition, hearing, speech production, breathing and sleeping. For this reason, they frequently undergo many surgical and non-surgical interventions between birth and 18 months of age, including orthodontic molding of the lip and nose, cleft lip and palate repair, and placement of tympanostomy tubes. Despite published care guidelines however, tremendous variation exists in care for infants with CL± P, nationally and internationally. This is in part due to a lack of standard outcome measures that can be applied in this age range across diverse treatments. Innovation: This project will contribute to outcomes development over the life span of persons born with CL± P and will provide an endpoint strategy to complement the efforts of the two largest cleft consortiums, Americleft and Eurocleft. We propose to develop novel outcomes to measure observable outcomes reported by parents. These methods are being explored by the FDA for qualifying instruments in device and drug development. The project will assess measurement properties of the parent measures in relation to clinical assessments agreed upon by leaders in the field. We address the entire endpoint strategy in clinical trials and augment existing measures with patient-centered outcomes, a high priority of research for NIH. The potential impact of the project is high, given our involvement of Americleft and Eurocleft leaders and our plans for involving leaders in the field from across the US and abroad in evaluation of the endpoint strategy we develop. Methods and Analysis: We will conduct Phase I interviews and focus group with parents and expert panels to obtain consensus on parent measure content and selection of clinical assessments. In Phase II, we will assess measurement properties of parents' measures and convene with expert panels to finalize parent measures and the endpoint strategy, including implementation of parent measures in clinical trials. Investigators: A multidisciplinary team of experienced researchers and clinicians (pediatrician, surgeons, clinical psychologists, clinical social workers biostatisticians, outcomes scientists) with a history of collaboration is well positioned to successfully accomplish the study aims.